Open Label Study of Subcutaneous Homoharringtonine (Omacetaxine Mepesuccinate) in Patients With Advanced CML

• Sponsor: Teva Branded Pharmaceutical Products R&D, Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00462943
• Collaborator: Cephalon (Industry), ChemGenex Pharmaceuticals (Industry)
• Enrollment: 100
• Locations: 29
• Arm: 1
• Actual Duration (Months): 75.7
• Patients Per Site: 3.4
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

A Phase II open-label trial of subcutaneous HHT (omacetaxine mepesuccinate) in the treatment of patients who are resistant to or intolerant to Tyrosine Kinase Inhibitors.

Condition or Disease	Intervention/Treatment	Phase
Chronic Myeloid Leukemia	Drug: Omacetaxine mepesuccinate	Phase 2

Detailed Description

This will be an open label, multicenter study of subcutaneous HHT (omacetaxine mepesuccinate) therapy of patients with chronic myeloid leukemia (CML) in chronic, accelerated, or blast phase who have failed or are intolerant to tyrosine kinase inhibitor therapy. Patients will be treated with induction course cycles consisting of subcutaneous (SC) HHT 1.25 mg/m² twice daily for 14 consecutive days every 28 days. Patients will be evaluated every 7 days with complete blood and platelet counts while undergoing induction therapy; the number of consecutive doses of HHT or intervals between subsequent cycles may be adjusted, as clinically indicated, according to guidelines provided in the treatment plan.

Study Design

• Study Type: Interventional
• Actual Enrollment: 100 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase II Open-Label Study of the Subcutaneous Administration of Homoharringtonine. (Omacetaxine Mepesuccinate; OMA) in the Treatment of Patients With Chronic Myeloid. Leukemia (CML) Who Have Failed or Are Intolerant to Tyrosine Kinase Inhibitor Therapy
• Actual Study Start Date: Mar 7, 2007
• Actual Primary Completion Date: Aug 4, 2009
• Actual Study Completion Date: Jun 27, 2013

Arms and Interventions

Arm

Intervention/Treatment

Experimental: OMA Omacetaxine mepesuccinate (OMA) Induction: 1.25mg/m^2 subcutaneously twice daily for 14 consecutive days, every 28 days for up to six cycles. Omacetaxine mepesuccinate (OMA) Maintenance: 1.25mg/m^2 subcutaneously twice daily for 7 consecutive days, every 28 days for up to 24 months.

Drug: Omacetaxine mepesuccinate; Induction: 1.25mg/m^2 subcutaneously twice daily for 14 consecutive days, every 28 days. Maintenance: 1.25mg/m^2 subcutaneously twice daily for 7 consecutive days, every 28 days. Response targets during induction vary by chronic myeloid leukemia (CML) subclass (chronic, accelerated, or blast phase). Participants will complete at least one cycle (14 days treatment of a 28 day cycle) of induction therapy before changing to maintenance therapy. Participants not demonstrating evidence of clinical response after 6 induction cycles will be considered for removal from the study.; Other Names: Homoharringtonine HHT Synribo OMA CGX-635

Outcome Measures

Primary Outcome Measures

1. Percentage of Participants Achieving an Overall Hematologic Response by Subpopulation and Total Population [Day 1 up to 6 months]

   Subpopulations reflect chronic myeloid leukemia (CML) phases at the time of enrollment: chronic, accelerated, and blast phase. Primary endpoints as adjudicated by the Data Monitoring Committee were used for the primary analyses. Overall hematologic response for chronic phase participants includes confirmed complete hematologic response (CHR). Overall hematologic response for accelerated or blast phase participants includes confirmed complete hematologic response (CHR), no evidence of leukemia (NEL), or return to chronic phase (RCP). Hematologic response must last >= 8 weeks to be considered meaningful. Response rates by disease phase were examined relative to an a priori value of 2.5% using a one-sided lower 95% exact binomial confidence limit. If the lower limit from the one-sided lower 95% confidence limit exceeds 2.5%, the observed response rate will have exceeded the minimum threshold required to demonstrate efficacy.

2. Percentage of Participants Achieving a Major Cytogenetic Response by Subpopulation and Total Population [Day 1 up to 9 months]

   Subpopulations reflect chronic myeloid leukemia (CML) phases at the time of enrollment: chronic, accelerated, and blast phase. Primary endpoints as adjudicated by the Data Monitoring Committee were used for the primary analyses. Major cytogenetic response includes complete or partial response. Both confirmed and unconfirmed major cytogenetic response is considered meaningful. Unconfirmed response is based on a single bone marrow cytogenetic evaluation for participants where a confirmatory evaluation is not available. Complete response shows 0% Philadelphia chromosome positive (Ph+) cells. A partial response shows >0% - 35% Ph+ cells. Response rates by disease phase were examined relative to an a priori value of 2.5% using a one-sided lower 95% exact binomial confidence limit. If the lower limit from the one-sided lower 95% confidence limit exceeds 2.5%, the observed response rate will have exceeded the minimum threshold required to demonstrate efficacy.

3. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total [up to 4 years]

   TEAE are any untoward events that were newly occurring or worsening from Baseline. Treatment related toxicity was considered by the investigator to be unrelated, possibly, probably or unknown related to study drug. Severity was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v3.0 on the following scale: Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe, Grade 4 = life-threatening, Grade 5 = death. A serious adverse event (SAE) is any untoward medical occurrence that is fatal or life-threatening; results in persistent or significant disability or incapacity; requires or prolongs in-patient hospitalization; is a congenital anomaly/birth defect in the offspring of a patient; and conditions not included in the above that may jeopardize the patient or may require intervention to prevent one of the outcomes listed above. A participant is only counted once in each category (at worst severity or strongest relationship).

Secondary Outcome Measures

1. Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+) [Day 1 up to Month 9]

   Cytogenetic response categories: Complete: 0% Ph+ cells Partial: >0%-35% Ph+ cells Minor: >35%-65% Ph+ cells Minimal: >65%-95% Ph+ cells No Response: >95% Ph+ cells Unevaluable: <20 metaphases were examined and/or response could not be assigned

2. Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene GUS [Day 1 up to Month 6]

   MMR is defined as a ratio of BCR-ABL/standard gene of less than 0.1% according to the international scale. BCR-ABL is a fusion gene of the breakpoint cluster region [BCR] gene and Abelson proto-oncogene [ABL] genes). This analysis used the standard gene GUS. Analysis was performed by quantitative reverse transcription polymerase chain reaction (qRT-PCR) of peripheral blood.

3. Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene ABL [Day 1 up to Month 6]

   MMR is defined as a ratio of BCR-ABL/standard gene of less than 0.1% according to the international scale. BCR-ABL is a fusion gene of the breakpoint cluster region [BCR] gene and Abelson proto-oncogene [ABL] genes). This analysis used the standard gene ABL. Analysis was performed by quantitative reverse transcription polymerase chain reaction (qRT-PCR) of peripheral blood.

4. Percentage of Participants in Each Hematologic Response Category [Day 1 up to Month 6]

   Complete Response (CHR) Chronic phase must last at least 8 weeks: WBC <10*10^9/liter, platelets <450*10^9/liter, myelocytes + metamyelocytes <5% in blood, no blasts or promyelocytes in blood, <20% basophils in peripheral blood, no extramedullary involvement. Accelerated and Blast phase must last at least 4 weeks: absolute neutrophil count 1.5*10^9/liter, platelets 100*10^9/liter, no blood blasts, bone marrow blasts <5%, no extramedullary disease. Partial Response - CHR plus one or more of the following: Persistence of splenomegaly with a reduction of ≥50% from pre-treatment Platelets > 450*10^9/L Presence of immature cells in the peripheral blood 5% to 25% blasts in the bone marrow If extra-medullary disease pre-treatment, reduction by ≥50% Hematologic Improvement - CHR, except allowing persistent thrombocytopenia (<100*10^9/L), and a few immature cells No evidence of leukemia: Morphologic leukemia-free state, defined as <5% bone marrow blasts.

5. Percentage of Participants With Extramedullary Disease (EMD) at Baseline Achieving a Clinical Response [Day 1 up to Month 9]

   Clinical response was defined by disease phase and based on evaluations by the independent Data Monitoring Committee (DMC). Chronic Phase subgroup: achieving a complete hematologic response and/or major cytogenetic response (complete cytogenetic response or partial cytogenetic response, confirmed or unconfirmed). Accelerated Phase and Blast Phase subgroups: achieving complete hematologic response, no evidence of leukemia, return to chronic phase, and/or major cytogenetic response (complete cytogenetic response or partial cytogenetic response, confirmed or unconfirmed).

6. Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL [Day 1 up to Month 9]

   Summarization is based on the best of the individual response assessments. Not assessable indicates that the participant either had no baseline assessment or the % mutation could not be determined in the post-baseline assessment(s).

7. Number of Treatment Cycles Needed to Achieve Best Hematologic Response [Day 1 up to Month 6]

   Induction therapy was administered for 14 consecutive days for each 28 days cycle, for up to 6 cycles. All treatment arms were given omacetaxine mepesuccinate via subcutaneous (SC) administration at 1.25 mg/m^2 twice a day (BID) for the 14 consecutive days.

8. Number of Treatment Cycles Needed to Achieve Best Cytogenetic Response [Day 1 up to Month 9]

9. Kaplan-Meier Estimates for Time to Onset of Best Hematologic Response [Day 1 up to Month 6]

   Time to onset was analyzed using Kaplan-Meier estimates. Participants who did not achieve a response are censored at their last visit day. Overall hematologic response for chronic phase participants includes confirmed complete hematologic response (CHR). Overall hematologic response for accelerated or blast phase participants includes confirmed complete hematologic response (CHR), no evidence of leukemia (NEL), or return to chronic phase (RCP). Hematologic response must last >= 8 weeks to be considered meaningful.

10. Kaplan-Meier Estimates for Time to Onset of Best Cytogenetic Response [Day 1 up to Month 9]

    Time to onset was analyzed using Kaplan-Meier estimates. Participants who did not achieve a response are censored at their last visit day. Major cytogenetic response includes complete or partial response. Both confirmed and unconfirmed major cytogenetic response is considered meaningful. Unconfirmed response is based on a single bone marrow cytogenetic evaluation for participants where a confirmatory evaluation is not available. Complete response shows 0% Philadelphia chromosome positive (Ph+) cells. A partial response shows >0% - 35% Ph+ cells.

11. Kaplan-Meier Estimates for Duration of Best Hematologic Response [up to four years]

    Duration of response is defined as the time from first reported date of hematologic response until the earliest date of objective evidence of disease progression, relapse or death. Data was censored at the last examination date for participants with ongoing response or participants who discontinued treatment for reasons other than adverse event, disease progression or death.

12. Kaplan-Meier Estimates for Duration of Best Cytogenetic Response [up to four years]

    Duration of response is defined as the time from first reported date of cytogenetic response until the earliest date of objective evidence of disease progression, relapse or death. Data was censored at the last examination date for participants with ongoing response or participants who discontinued treatment for reasons other than adverse event, disease progression or death.

13. Kaplan-Meier Estimates for Time to Disease Progression [up to 4 years]

    Time to disease progression is defined as the time from the initiation of treatment until the onset date of death, the development of CML accelerated phase or blast phase, or the loss of complete hematologic response or major cytogenetic response, whichever came first. Participants were censored only if they did not have progression or if they discontinued treatment for reasons other than AE, progression or death.

14. Kaplan-Meier Estimates for Overall Survival [up to 4 years]

    Overall survival is defined as the time from the initiation of treatment until death from any cause or the last day of participant contact or evaluation for participants that were lost to follow-up. Participants were censored t the last recorded contract or evaluation when a participant was alive at time of analysis. A quarterly phone survey was conducted to collect survival data for participants who discontinued from the study.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Male or female patients, age 18 years or older

• Philadelphia chromosome (Ph) positive chronic myelogenous leukemia in either chronic, accelerated, or blast phase

• Patients will have either failed, demonstrated intolerance, or a combination of prior failure and intolerance, to prior treatments with at least two tyrosine kinase inhibitors (TKI's). Failure of TKI treatment may either be primary (never achieved a response) or secondary resistance (loss of response).

• Acceptable Renal and Liver Function

• Eastern Cooperative Oncology Group (ECOG) performance status 0-2.

• Sexually active patients and their partners must use an effective double barrier method of contraception

Exclusion Criteria:

• New York Heart Association classification (NYHA) class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition

• Myocardial infarction in the previous 12 weeks.

• Other concurrent illness which would preclude study conduct and assessment

• uncontrolled and active infection, and positive HIV or positive HTLV I/II status, whether on treatment or not.

• Pregnant or lactating.

• Any medical or psychiatric condition, which may compromise the ability to give written informed consent or to comply with the study protocol.

• Lymphoid Ph+ blast crisis

• Patient is enrolled in another clinical investigation within 30 days of enrollment or is receiving another investigational agent

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Teva Investigational Site 303	Los Angeles	California	United States	90033
2	Teva Investigational Site 308	Beech Grove	Indiana	United States	46107
3	Teva Investigational Site 311	Baltimore	Maryland	United States	21201
4	Teva Investigational Site 302	Bronx	New York	United States	10466
5	Teva Investigational Site 305	Buffalo	New York	United States	14263
6	Teva Investigational Site 310	Philadelphia	Pennsylvania	United States	19111
7	Teva Investigational Site 301	Houston	Texas	United States	77030
8	Teva Investigational Site 314	Seattle	Washington	United States	98109
9	Teva Investigational Site 313	Montreal		Canada	H3a 1a1
10	Teva Investigational Site 309	Toronto		Canada	M5G 2M9
11	Teva Investigational Site 329	Bordeaux		France	33076
12	Teva Investigational Site 321	Le Chesnay Cedex		France	78157
13	Teva Investigational Site 322	Lille		France	59000
14	Teva Investigational Site 320	Lyon Cedex 03		France	69437
15	Teva Investigational Site 324	Nice		France	06202
16	Teva Investigational Site 328	Paris		France	75475
17	Teva Investigational Site 323	Poitiers Cedex		France	86021
18	Teva Investigational Site 327	Strasbourg		France	67100
19	Teva Investigational Site 325	Toulouse		France	31059
20	Teva Investigational Site 331	Berlin		Germany	10117
21	Teva Investigational Site 330	Mannheim		Germany	68169
22	Teva Investigational Site 350	Budapest		Hungary	1096
23	Teva Investigational Site 371	Hyderabad		India	500082
24	Teva Investigational Site 370	Mumbai		India	400 014
25	Teva Investigational Site 390	Bologna		Italy	41038
26	Teva Investigational Site 360	Gdansk		Poland	80-952
27	Teva Investigational Site 361	Warszawa		Poland	02776
28	Teva Investigational Site 380	Singapore		Singapore	169608
29	Teva Investigational Site 340	London		United Kingdom	W12 0HS

Sponsors and Collaborators

• Teva Branded Pharmaceutical Products R&D, Inc.
• Cephalon
• ChemGenex Pharmaceuticals

Investigators

• Principal Investigator: Jorge Cortes, MD, M.D. Anderson Cancer Center

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Teva Branded Pharmaceutical Products R&D, Inc.
• ClinicalTrials.gov Identifier: NCT00462943
• Other Study ID Numbers: CGX-635-CML-203; 2007-001286-15
• First Posted: Apr 19, 2007
• Last Update Posted: Dec 28, 2021
• Last Verified: Dec 1, 2021

• Keywords provided by Teva Branded Pharmaceutical Products R&D, Inc.: CML; HHT; Homoharringtonine; Omacetaxine
• Additional relevant MeSH terms: Leukemia; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Homoharringtonine

Study Results

Participant Flow

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Period Title: Overall Study
STARTED	46	31	23
COMPLETED	1	1	0
NOT COMPLETED	45	30	23

Baseline Characteristics

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Total of all reporting groups
Overall Participants	46	31	23	100
Age (years) [Median (Full Range) ]
Median (Full Range) [years]	58	56	57	57
Sex: Female, Male (Count of Participants)
Female	20 43.5%	12 38.7%	9 39.1%	41 41%
Male	26 56.5%	19 61.3%	14 60.9%	59 59%
Race/Ethnicity, Customized (participants) [Number]
Caucasian	31 67.4%	17 54.8%	12 52.2%	60 60%
Black	2 4.3%	4 12.9%	5 21.7%	11 11%
Hispanic	3 6.5%	2 6.5%	1 4.3%	6 6%
Asian	7 15.2%	7 22.6%	4 17.4%	18 18%
Other	3 6.5%	1 3.2%	1 4.3%	5 5%
Height (centimeter) [Median (Full Range) ]
Median (Full Range) [centimeter]	167.7	167.5	171.0	167.6
Weight (kilograms) [Median (Full Range) ]
Median (Full Range) [kilograms]	79.5	67.4	74.0	74.2
Body Surface Area (BSA) (meters^2) [Median (Full Range) ]
Median (Full Range) [meters^2]	1.9	1.7	1.9	1.9
New York Heart Association (NYHA) Classification (participants) [Number]
Class I	44 95.7%	29 93.5%	23 100%	96 96%
Class II	2 4.3%	0 0%	0 0%	2 2%
Class III	0 0%	0 0%	0 0%	0 0%
Class IV	0 0%	0 0%	0 0%	0 0%
Not available	0 0%	2 6.5%	0 0%	2 2%
Eastern Cooperative Oncology Group Performance Status (participants) [Number]
Grade 0	27 58.7%	7 22.6%	6 26.1%	40 40%
Grade 1	17 37%	19 61.3%	11 47.8%	47 47%
Grade 2	2 4.3%	5 16.1%	5 21.7%	12 12%
Grade 3	0 0%	0 0%	1 4.3%	1 1%
Time from Initial Chronic Myeloid Leukemia (CML) Diagnosis (months) [Median (Full Range) ]
Median (Full Range) [months]	74.4	83.5	68.7	74.0

Outcome Measures

1. Primary Outcome

Title	Percentage of Participants Achieving an Overall Hematologic Response by Subpopulation and Total Population
Description	Subpopulations reflect chronic myeloid leukemia (CML) phases at the time of enrollment: chronic, accelerated, and blast phase. Primary endpoints as adjudicated by the Data Monitoring Committee were used for the primary analyses. Overall hematologic response for chronic phase participants includes confirmed complete hematologic response (CHR). Overall hematologic response for accelerated or blast phase participants includes confirmed complete hematologic response (CHR), no evidence of leukemia (NEL), or return to chronic phase (RCP). Hematologic response must last >= 8 weeks to be considered meaningful. Response rates by disease phase were examined relative to an a priori value of 2.5% using a one-sided lower 95% exact binomial confidence limit. If the lower limit from the one-sided lower 95% confidence limit exceeds 2.5%, the observed response rate will have exceeded the minimum threshold required to demonstrate efficacy.
Time Frame	Day 1 up to 6 months

Outcome Measure Data

Analysis Population Description

Intent to treat population

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	46	31	23	100
Number (95% Confidence Interval) [percentage of participants]	67.4 146.5%	25.8 83.2%	8.7 37.8%	41.0 41%

2. Primary Outcome

Title	Percentage of Participants Achieving a Major Cytogenetic Response by Subpopulation and Total Population
Description	Subpopulations reflect chronic myeloid leukemia (CML) phases at the time of enrollment: chronic, accelerated, and blast phase. Primary endpoints as adjudicated by the Data Monitoring Committee were used for the primary analyses. Major cytogenetic response includes complete or partial response. Both confirmed and unconfirmed major cytogenetic response is considered meaningful. Unconfirmed response is based on a single bone marrow cytogenetic evaluation for participants where a confirmatory evaluation is not available. Complete response shows 0% Philadelphia chromosome positive (Ph+) cells. A partial response shows >0% - 35% Ph+ cells. Response rates by disease phase were examined relative to an a priori value of 2.5% using a one-sided lower 95% exact binomial confidence limit. If the lower limit from the one-sided lower 95% confidence limit exceeds 2.5%, the observed response rate will have exceeded the minimum threshold required to demonstrate efficacy.
Time Frame	Day 1 up to 9 months

Outcome Measure Data

Analysis Population Description

Intent to treat population

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	46	31	23	100
Number (95% Confidence Interval) [percentage of participants]	21.7 47.2%	3.2 10.3%	0 0%	11 11%

3. Secondary Outcome

Title	Percentage of Participants in Each Cytogenetic Response Category Representing the Degree of Suppression of the Philadelphia Chromosome (Ph+)
Description	Cytogenetic response categories: Complete: 0% Ph+ cells Partial: >0%-35% Ph+ cells Minor: >35%-65% Ph+ cells Minimal: >65%-95% Ph+ cells No Response: >95% Ph+ cells Unevaluable: <20 metaphases were examined and/or response could not be assigned
Time Frame	Day 1 up to Month 9

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	46	31	23	100
Complete	4.3 9.3%	0 0%	0 0%	2.0 2%
Partial	17.4 37.8%	3.2 10.3%	0 0%	9.0 9%
Minor	8.7 18.9%	9.7 31.3%	0 0%	7.0 7%
Minimal	6.5 14.1%	6.5 21%	4.3 18.7%	6.0 6%
No Response	39.1 85%	61.3 197.7%	30.4 132.2%	44.0 44%
Unevaluable	23.9 52%	19.4 62.6%	65.2 283.5%	32.0 32%

4. Secondary Outcome

Title	Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene GUS
Description	MMR is defined as a ratio of BCR-ABL/standard gene of less than 0.1% according to the international scale. BCR-ABL is a fusion gene of the breakpoint cluster region [BCR] gene and Abelson proto-oncogene [ABL] genes). This analysis used the standard gene GUS. Analysis was performed by quantitative reverse transcription polymerase chain reaction (qRT-PCR) of peripheral blood.
Time Frame	Day 1 up to Month 6

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants who had evaluable samples. Participants with no data for these analyses either had degraded samples or the samples were missing.

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	22	18	4	44
Number (95% Confidence Interval) [percentage of participants]	13.6 29.6%	0 0%	0 0%	6.8 6.8%

5. Secondary Outcome

Title	Percentage of Participants With Major Molecular Response (MMR) Representing the Degree of Suppression of BCR-ABL Transcript Levels Using the Housekeeping Gene ABL
Description	MMR is defined as a ratio of BCR-ABL/standard gene of less than 0.1% according to the international scale. BCR-ABL is a fusion gene of the breakpoint cluster region [BCR] gene and Abelson proto-oncogene [ABL] genes). This analysis used the standard gene ABL. Analysis was performed by quantitative reverse transcription polymerase chain reaction (qRT-PCR) of peripheral blood.
Time Frame	Day 1 up to Month 6

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants who had evaluable samples. Participants with no data for these analyses either had degraded samples or the samples were missing.

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	38	23	11	72
Number (95% Confidence Interval) [percentage of participants]	10.5 22.8%	4.3 13.9%	0 0%	6.9 6.9%

6. Secondary Outcome

Title	Percentage of Participants in Each Hematologic Response Category
Description	Complete Response (CHR) Chronic phase must last at least 8 weeks: WBC <10*10^9/liter, platelets <450*10^9/liter, myelocytes + metamyelocytes <5% in blood, no blasts or promyelocytes in blood, <20% basophils in peripheral blood, no extramedullary involvement. Accelerated and Blast phase must last at least 4 weeks: absolute neutrophil count 1.5*10^9/liter, platelets 100*10^9/liter, no blood blasts, bone marrow blasts <5%, no extramedullary disease. Partial Response - CHR plus one or more of the following: Persistence of splenomegaly with a reduction of ≥50% from pre-treatment Platelets > 450*10^9/L Presence of immature cells in the peripheral blood 5% to 25% blasts in the bone marrow If extra-medullary disease pre-treatment, reduction by ≥50% Hematologic Improvement - CHR, except allowing persistent thrombocytopenia (<100*10^9/L), and a few immature cells No evidence of leukemia: Morphologic leukemia-free state, defined as <5% bone marrow blasts.
Time Frame	Day 1 up to Month 6

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	46	31	23	100
Complete response	67.4 146.5%	19.4 62.6%	8.7 37.8%	39.0 39%
Partial response	0 0%	3.2 10.3%	0 0%	1.0 1%
Hematologic improvement	0 0%	9.7 31.3%	4.3 18.7%	4.0 4%
Return to chronic phase	NA NaN	6.5 21%	0 0%	2.0 2%
No evidence of leukemia	NA NaN	0 0%	0 0%	0 0%
No response	21.7 47.2%	58.1 187.4%	78.3 340.4%	46.0 46%
Unevaluable	10.9 23.7%	3.2 10.3%	8.7 37.8%	8.0 8%

7. Secondary Outcome

Title	Percentage of Participants With Extramedullary Disease (EMD) at Baseline Achieving a Clinical Response
Description	Clinical response was defined by disease phase and based on evaluations by the independent Data Monitoring Committee (DMC). Chronic Phase subgroup: achieving a complete hematologic response and/or major cytogenetic response (complete cytogenetic response or partial cytogenetic response, confirmed or unconfirmed). Accelerated Phase and Blast Phase subgroups: achieving complete hematologic response, no evidence of leukemia, return to chronic phase, and/or major cytogenetic response (complete cytogenetic response or partial cytogenetic response, confirmed or unconfirmed).
Time Frame	Day 1 up to Month 9

Outcome Measure Data

Analysis Population Description

Intent to treat population of study participants who had extramedullary disease at baseline

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	0	0	2
Clinical response	0 0%
Unevaluable	50 108.7%

8. Secondary Outcome

Title	Percentage of Participants With the Largest Percentage Reduction From Baseline of T315I Mutated BCR-ABL
Description	Summarization is based on the best of the individual response assessments. Not assessable indicates that the participant either had no baseline assessment or the % mutation could not be determined in the post-baseline assessment(s).
Time Frame	Day 1 up to Month 9

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants with post-baseline assessment of T315I mutated BCR ABL

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	38	23	11	72
100% reduction	0 0%	0 0%	0 0%	0 0%
75-99% reduction	0 0%	0 0%	0 0%	0 0%
50-74% reduction	0 0%	0 0%	0 0%	0 0%
25-49% reduction	0 0%	0 0%	9.1 39.6%	1.4 1.4%
1-24% reduction	0 0%	0 0%	0 0%	0 0%
0% reduction	78.9 171.5%	91.3 294.5%	63.3 275.2%	80.6 80.6%
Not assessable	21.1 45.9%	8.7 28.1%	27.3 118.7%	18.1 18.1%

9. Secondary Outcome

Title	Number of Treatment Cycles Needed to Achieve Best Hematologic Response
Description	Induction therapy was administered for 14 consecutive days for each 28 days cycle, for up to 6 cycles. All treatment arms were given omacetaxine mepesuccinate via subcutaneous (SC) administration at 1.25 mg/m^2 twice a day (BID) for the 14 consecutive days.
Time Frame	Day 1 up to Month 6

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants who had a response to treatment

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	31	8	2	41
Median (Full Range) [treatment cycles]	1.0	2.0	2.0	1.0

10. Secondary Outcome

Title	Number of Treatment Cycles Needed to Achieve Best Cytogenetic Response
Time Frame	Day 1 up to Month 9

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants who had a cytogenetic response

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	17	6	1	24
Median (Full Range) [treatment cycles]	2.0	1.5	1.0	2.0

11. Secondary Outcome

Title	Kaplan-Meier Estimates for Time to Onset of Best Hematologic Response
Description	Time to onset was analyzed using Kaplan-Meier estimates. Participants who did not achieve a response are censored at their last visit day. Overall hematologic response for chronic phase participants includes confirmed complete hematologic response (CHR). Overall hematologic response for accelerated or blast phase participants includes confirmed complete hematologic response (CHR), no evidence of leukemia (NEL), or return to chronic phase (RCP). Hematologic response must last >= 8 weeks to be considered meaningful.
Time Frame	Day 1 up to Month 6

Outcome Measure Data

Analysis Population Description

Intent to treat population.

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	46	31	23	100
Median (95% Confidence Interval) [months]	1.38	NA	NA	5.03

12. Secondary Outcome

Title	Kaplan-Meier Estimates for Time to Onset of Best Cytogenetic Response
Description	Time to onset was analyzed using Kaplan-Meier estimates. Participants who did not achieve a response are censored at their last visit day. Major cytogenetic response includes complete or partial response. Both confirmed and unconfirmed major cytogenetic response is considered meaningful. Unconfirmed response is based on a single bone marrow cytogenetic evaluation for participants where a confirmatory evaluation is not available. Complete response shows 0% Philadelphia chromosome positive (Ph+) cells. A partial response shows >0% - 35% Ph+ cells.
Time Frame	Day 1 up to Month 9

Outcome Measure Data

Analysis Population Description

Intent to treat population.

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	46	31	23
Median (95% Confidence Interval) [months]	NA	NA	NA

13. Primary Outcome

Title	Number of Participants With Treatment-Emergent Adverse Events (TEAEs) by Subpopulation and Total
Description	TEAE are any untoward events that were newly occurring or worsening from Baseline. Treatment related toxicity was considered by the investigator to be unrelated, possibly, probably or unknown related to study drug. Severity was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v3.0 on the following scale: Grade 1 = mild, Grade 2 = moderate, Grade 3 = severe, Grade 4 = life-threatening, Grade 5 = death. A serious adverse event (SAE) is any untoward medical occurrence that is fatal or life-threatening; results in persistent or significant disability or incapacity; requires or prolongs in-patient hospitalization; is a congenital anomaly/birth defect in the offspring of a patient; and conditions not included in the above that may jeopardize the patient or may require intervention to prevent one of the outcomes listed above. A participant is only counted once in each category (at worst severity or strongest relationship).
Time Frame	up to 4 years

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	46	31	23	100
>=1 TEAE	46 100%	31 100%	23 100%	100 100%
>= 1 SAE	26 56.5%	19 61.3%	18 78.3%	63 63%
Worst severity: Grade 1	1 2.2%	0 0%	0 0%	1 1%
Worst severity: Grade 2	4 8.7%	4 12.9%	2 8.7%	10 10%
Worst severity: Grade 3	18 39.1%	6 19.4%	4 17.4%	28 28%
Worst severity: Grade 4	17 37%	15 48.4%	6 26.1%	38 38%
Worst severity: Grade 5	6 13%	6 19.4%	11 47.8%	23 23%
Relation to drug: Unrelated	4 8.7%	4 12.9%	7 30.4%	15 15%
Relation to drug: Possibly	5 10.9%	11 35.5%	7 30.4%	23 23%
Relation to drug: Probably	36 78.3%	14 45.2%	8 34.8%	58 58%
Relation to drug: Unknown	1 2.2%	2 6.5%	1 4.3%	4 4%
With hematologic toxicity	36 78.3%	22 71%	13 56.5%	71 71%
Discontinued treatment due to AE	10 21.7%	11 35.5%	6 26.1%	27 27%
Deaths during study or follow-up	35 76.1%	25 80.6%	21 91.3%	71 71%
Deaths during study (outcome of SAE)	6 13%	6 19.4%	11 47.8%	23 23%

14. Secondary Outcome

Title	Kaplan-Meier Estimates for Duration of Best Hematologic Response
Description	Duration of response is defined as the time from first reported date of hematologic response until the earliest date of objective evidence of disease progression, relapse or death. Data was censored at the last examination date for participants with ongoing response or participants who discontinued treatment for reasons other than adverse event, disease progression or death.
Time Frame	up to four years

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants who had a response

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	31	8	2
Median (Full Range) [months]	7.01	5.47	2.67

15. Secondary Outcome

Title	Kaplan-Meier Estimates for Duration of Best Cytogenetic Response
Description	Duration of response is defined as the time from first reported date of cytogenetic response until the earliest date of objective evidence of disease progression, relapse or death. Data was censored at the last examination date for participants with ongoing response or participants who discontinued treatment for reasons other than adverse event, disease progression or death.
Time Frame	up to four years

Outcome Measure Data

Analysis Population Description

Intent to treat population of participants who had a response

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	10	1	0
Median (Full Range) [months]	6.01	0.07

16. Secondary Outcome

Title	Kaplan-Meier Estimates for Time to Disease Progression
Description	Time to disease progression is defined as the time from the initiation of treatment until the onset date of death, the development of CML accelerated phase or blast phase, or the loss of complete hematologic response or major cytogenetic response, whichever came first. Participants were censored only if they did not have progression or if they discontinued treatment for reasons other than AE, progression or death.
Time Frame	up to 4 years

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	46	31	23	100
Median (95% Confidence Interval) [months]	7.50	4.84	2.04	4.38

17. Secondary Outcome

Title	Kaplan-Meier Estimates for Overall Survival
Description	Overall survival is defined as the time from the initiation of treatment until death from any cause or the last day of participant contact or evaluation for participants that were lost to follow-up. Participants were censored t the last recorded contract or evaluation when a participant was alive at time of analysis. A quarterly phone survey was conducted to collect survival data for participants who discontinued from the study.
Time Frame	up to 4 years

Outcome Measure Data

Analysis Population Description

Intent to treat

Arm/Group Title	CML: Chronic Phase	CML: Accelerated Phase	CML: Blast Phase	Total Participants
Arm/Group Description	Study participants with chronic myeloid leukemia (CML) in the chronic phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the accelerated phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Study participants with chronic myeloid leukemia (CML) in the blast phase at the time of enrollment. Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.	Treatment included induction therapy of subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
Measure Participants	46	31	23	100
Median (95% Confidence Interval) [months]	33.91	17.27	3.52	17.27

Adverse Events

Time Frame	up to 4 years
Arm/Group Title	Omacetaxine
Arm/Group Description	Treatment was the same for all cohorts: induction therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 14 consecutive days every 28 (±3) days for up to 6 cycles. Maintenance therapy was subcutaneous (SC) administration of omacetaxine at 1.25 mg/m^2 twice a day (BID), administered for 7 consecutive days every 28 (±3) days for up to 24 months.
All Cause Mortality
	Omacetaxine
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Omacetaxine
	Affected / at Risk (%)	# Events
Total	63/100 (63%)
Blood and lymphatic system disorders
Anaemia	3/100 (3%)	3
Anaemia haemolytic autoimmune	1/100 (1%)	1
Bone marrow failure	5/100 (5%)	6
Febrile bone marrow aplasia	2/100 (2%)	2
Febrile neutropenia	9/100 (9%)	14
Leukocytosis	1/100 (1%)	1
Neutropenia	1/100 (1%)	1
Pancytopenia	2/100 (2%)	2
Thrombocytopenia	6/100 (6%)	10
Cardiac disorders
Acute coronary syndrome	1/100 (1%)	1
Atrial fibrillation	1/100 (1%)	1
Cardiac failure congestive	1/100 (1%)	1
Pericardial effusion	1/100 (1%)	1
Pericarditis	1/100 (1%)	1
Tachycardia	1/100 (1%)	1
Ear and labyrinth disorders
Vertigo	1/100 (1%)	1
Gastrointestinal disorders
Diarrhoea	4/100 (4%)	4
Gastrointestinal haemorrhage	2/100 (2%)	2
Pancreatitis	1/100 (1%)	1
Stomatitis	2/100 (2%)	2
General disorders
Chest pain	1/100 (1%)	1
Death	1/100 (1%)	1
Disease progression	8/100 (8%)	8
Fatigue	2/100 (2%)	2
Localised oedema	1/100 (1%)	1
Multi-organ failure	1/100 (1%)	1
Pyrexia	4/100 (4%)	5
Hepatobiliary disorders
Cholelithiasis	1/100 (1%)	1
Infections and infestations
Bacteraemia	1/100 (1%)	4
Catheter sepsis	2/100 (2%)	2
Injection site infection	1/100 (1%)	1
Lung infection	2/100 (2%)	2
Pneumonia	5/100 (5%)	5
Sepsis	3/100 (3%)	4
Tonsillitis	1/100 (1%)	2
Viral infection	1/100 (1%)	1
Injury, poisoning and procedural complications
Post procedural haemorrhage	1/100 (1%)	1
Subdural haematoma	1/100 (1%)	1
Transfusion reaction	1/100 (1%)	2
Metabolism and nutrition disorders
Dehydration	1/100 (1%)	1
Failure to thrive	2/100 (2%)	2
Hypercalcaemia	3/100 (3%)	3
Musculoskeletal and connective tissue disorders
Back pain	1/100 (1%)	1
Neck pain	1/100 (1%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Blast cell crisis	1/100 (1%)	1
Chronic myeloid leukaemia	2/100 (2%)	2
Leukaemia	2/100 (2%)	2
Metastatic neoplasm	1/100 (1%)	1
Myelodysplastic syndrome	1/100 (1%)	1
Nervous system disorders
Cerebral haemorrhage	3/100 (3%)	3
Cerebral ischaemia	1/100 (1%)	1
Convulsion	1/100 (1%)	1
Psychiatric disorders
Mood altered	1/100 (1%)	1
Respiratory, thoracic and mediastinal disorders
Acute pulmonary oedema	1/100 (1%)	1
Dyspnoea	1/100 (1%)	1
Pulmonary embolism	1/100 (1%)	1
Pulmonary haemorrhage	1/100 (1%)	1
Vascular disorders
Hypotension	1/100 (1%)	1
Other (Not Including Serious) Adverse Events
	Omacetaxine
	Affected / at Risk (%)	# Events
Total	99/100 (99%)
Blood and lymphatic system disorders
Anaemia	51/100 (51%)	132
Bone marrow failure	6/100 (6%)	7
Febrile neutropenia	17/100 (17%)	24
Leukocytosis	7/100 (7%)	10
Leukopenia	13/100 (13%)	55
Lymphopenia	8/100 (8%)	20
Neutropenia	31/100 (31%)	114
Thrombocytopenia	62/100 (62%)	192
Cardiac disorders
Tachycardia	5/100 (5%)	6
Gastrointestinal disorders
Abdominal distension	5/100 (5%)	5
Abdominal pain	15/100 (15%)	21
Abdominal pain upper	7/100 (7%)	7
Constipation	11/100 (11%)	12
Diarrhoea	42/100 (42%)	71
Dyspepsia	7/100 (7%)	7
Gingival bleeding	5/100 (5%)	5
Nausea	29/100 (29%)	44
Stomatitis	10/100 (10%)	16
Vomiting	17/100 (17%)	22
General disorders
Asthenia	19/100 (19%)	46
Chills	10/100 (10%)	13
Disease progression	10/100 (10%)	11
Fatigue	24/100 (24%)	37
Injection site erythema	11/100 (11%)	34
Injection site rash	5/100 (5%)	5
Injection site reaction	5/100 (5%)	6
Mucosal inflammation	5/100 (5%)	5
Oedema peripheral	14/100 (14%)	22
Pyrexia	28/100 (28%)	50
Infections and infestations
Bronchitis	8/100 (8%)	8
Cellulitis	6/100 (6%)	8
Pneumonia	13/100 (13%)	15
Upper respiratory tract infection	9/100 (9%)	13
Urinary tract infection	6/100 (6%)	7
Injury, poisoning and procedural complications
Contusion	8/100 (8%)	9
Investigations
Alanine aminotransferase increased	6/100 (6%)	6
Metabolism and nutrition disorders
Anorexia	14/100 (14%)	15
Hyperuricaemia	8/100 (8%)	9
Hypokalaemia	8/100 (8%)	10
Musculoskeletal and connective tissue disorders
Arthralgia	10/100 (10%)	11
Back pain	7/100 (7%)	7
Bone pain	9/100 (9%)	10
Pain in extremity	15/100 (15%)	18
Nervous system disorders
Dizziness	6/100 (6%)	8
Headache	20/100 (20%)	27
Psychiatric disorders
Insomnia	7/100 (7%)	9
Respiratory, thoracic and mediastinal disorders
Cough	13/100 (13%)	22
Dyspnoea	8/100 (8%)	10
Epistaxis	17/100 (17%)	19
Pharyngolaryngeal pain	9/100 (9%)	9
Skin and subcutaneous tissue disorders
Alopecia	7/100 (7%)	7
Rash	7/100 (7%)	8
Vascular disorders
Haematoma	5/100 (5%)	12
Hypertension	6/100 (6%)	8

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

Sponsor has the right 60 days before submission for publication to review/provide comments. If the Sponsor's review shows that potentially patentable subject matter would be disclosed, publication or public disclosure shall be delayed for up to 90 additional days in order for the Sponsor, or Sponsor's designees, to file the necessary patent applications. In multicenter trials, each PI will postpone single center publications until after disclosure or publication of multicenter data.